Rare Daily Staff
The U.S. Food and Drug Administration has expanded the approved use of Amgen’s Nplate to include the treatment of pediatric patients one year of age and older with immune thrombocytopenia.
The approval is for use of Nplate for at least six months in children who have had an insufficient response to corticosteroids, immunoglobulins, or had their spleens removed.
Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder characterized by low platelet counts in the blood and impaired platelet production. Platelets are specialized blood cells that prevent and stop bleeding during clotting. As the platelet count falls, the risk of developing bleeding symptoms increases.
Nplate is a thrombopoietin (TPO) receptor agonist that mimics the body’s natural TPO and is designed to increase platelet counts in patients with chronic ITP.
“Children with ITP are at risk for serious bleeding events and spontaneous bruising due to low platelet counts, which can be worrying for these young patients and their parents. Currently, these patients have a limited number of treatment options, especially for those with refractory disease,” said Michael Tarantino, president of the Bleeding and Clotting Disorders Institute. “Today’s approval of Nplate offers new hope to the pediatric ITP community as it provides children with a new treatment option that may help to maintain safe platelet counts.”
The FDA based its approval on two placebo-controlled studies: a phase 3 and phase 1/2 – evaluating the safety and efficacy of Nplate in pediatric patients. In the phase 3 study, published in The Lancet, rates of overall platelet response were increased with the Nplate group (71 percent) compared with placebo (20 percent). Additionally, durable platelet response occurred more frequently with Nplate (52 percent) compared with placebo (10 percent).
In the two placebo-controlled trials, adverse reactions with an incidence of greater than 25 percent in the Nplate arm were contusion, upper respiratory tract infection, and oropharyngeal pain.
Nplate is also approved in 67 countries, including Canada, Australia and Japan.
December 18, 2018
Photo: Michael Tarantino, president of the Bleeding and Clotting Disorders Institute